CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes

Cystic fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in people with the most prevalent F508del mutation. Ho...

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Detalles Bibliográficos
Autores principales: Lefferts, Juliet W., Bierlaagh, Marlou C., Kroes, Suzanne, Nieuwenhuijze, Natascha D. A., Sonneveld van Kooten, Heleen N., Niemöller, Paul J., Verburg, Tibo F., Janssens, Hettie M., Muilwijk, Danya, van Beuningen, Sam F. B., van der Ent, Cornelis K., Beekman, Jeffrey M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10572896/
https://www.ncbi.nlm.nih.gov/pubmed/37833986
http://dx.doi.org/10.3390/ijms241914539

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10572896/
https://www.ncbi.nlm.nih.gov/pubmed/37833986
http://dx.doi.org/10.3390/ijms241914539

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